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ClinicalTrials.gov public records Last synced Nov 14, 2026, 6:13 PM EST

All trial information on this site comes from the official ClinicalTrials.gov public API (clinicaltrials.gov/api/v2), run by the U.S. National Library of Medicine. We only display that public information; we do not add opinions, endorsements, or any other intent. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 512 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Pediatric: Genetic Syndrome Clear all

Conditions: Duchenne Muscular Dystrophy
Interventions: delandistrogene moxeparvovec, placebo; Genetic
Lead sponsor: Sarepta Therapeutics, Inc.; Industry
Eligibility: 4 Years to 7 Years · Male only
Enrollment: 126 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2024
U.S. locations: 23
States / cities: Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 20 more

Conditions: Mucopolysaccharidosis II
Interventions: Not listed
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: Male only
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2022
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Neurofibroma, Plexiform, Neurofibromatosis Type I
Interventions: tipifarnib, placebo; Drug · Other
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 3 Years to 25 Years
Enrollment: 62 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2009
U.S. locations: 11
States / cities: Birmingham, Alabama • Los Angeles, California • Chicago, Illinois + 8 more

Conditions: Pulmonary Hypertension
Interventions: Not listed
Lead sponsor: Akron Children's Hospital; Other
Eligibility: 7 Years to 21 Years
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2013
U.S. locations: 1
States / cities: Akron, Ohio

Conditions: Neuroblastoma, Osteosarcoma
Interventions: iC9.GD2.CAR.IL-15 T-cells, Cyclophosphamide, Fludarabine; Biological · Drug
Lead sponsor: UNC Lineberger Comprehensive Cancer Center; Other
Eligibility: 18 Months and older
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2044
U.S. locations: 2
States / cities: Atlanta, Georgia • Chapel Hill, North Carolina

Conditions: Menkes Disease
Interventions: Droxidopa Oral Product, Placebo Control; Drug · Other
Lead sponsor: Stephen G. Kaler; Other
Eligibility: 7 Years to 17 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2030
U.S. locations: 1
States / cities: New York, New York

Conditions: Congenital Hemophilia A
Interventions: rVIII-SingleChain; Biological
Lead sponsor: CSL Behring; Industry
Eligibility: Up to 11 Years · Male only
Enrollment: 84 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2015
U.S. locations: 2
States / cities: Aurora, Colorado • Chicago, Illinois

Conditions: Sickle Cell Disease
Interventions: Morphine, Methadone; Drug
Lead sponsor: Washington University School of Medicine; Other
Eligibility: 7 Years to 40 Years
Enrollment: 47 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2010
U.S. locations: 1
States / cities: St Louis, Missouri

Conditions: Tourette Syndrome in Children, Tourette Syndrome in Adolescence
Interventions: T92, Placebo; Dietary Supplement
Lead sponsor: Tasly Pharmaceuticals, Inc.; Industry
Eligibility: 6 Years to 17 Years
Enrollment: 150 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2024
U.S. locations: 1
States / cities: New Haven, Connecticut

Conditions: Von Willebrand Disease, Blood Coagulation Disorders, Blood Platelet Disorders, Hematologic Disease
Interventions: Blood coagulation Factor VIII and vWF, human; Drug
Lead sponsor: CSL Behring; Industry
Eligibility: Not listed
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2006
U.S. locations: 1
States / cities: Milwaukee, Wisconsin

Conditions: Duchenne Muscular Dystrophy
Interventions: delandistrogene moxeparvovec; Genetic
Lead sponsor: Sarepta Therapeutics, Inc.; Industry
Eligibility: 2 Years and older · Male only
Enrollment: 83 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2028
U.S. locations: 12
States / cities: Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 9 more

Conditions: Angelman Syndrome
Interventions: No intervention, GTX-102; Other · Drug
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Eligibility: 1 Year to 64 Years
Enrollment: 55 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2026
U.S. locations: 8
States / cities: Los Angeles, California • Chicago, Illinois • Baltimore, Maryland + 5 more

Conditions: Sickle Cell Disease, Vaso-occlusive Pain Episode
Interventions: L-arginine, L-arginine Loading Dose, Placebo; Drug · Other
Lead sponsor: Emory University; Other
Eligibility: 3 Years to 21 Years
Enrollment: 108 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2021
U.S. locations: 3
States / cities: Atlanta, Georgia

Conditions: Sickle Cell Disease, Hematopoetic Stem Cell Transplant, Haploidentical Hematopoietic Stem Cell Transplant, Haploidentical Stem Cell Transplantation, Graft Failure
Interventions: Ruxolitinib; Drug
Lead sponsor: Arkansas Children's Hospital Research Institute; Other
Eligibility: 12 Years to 45 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2029
U.S. locations: 4
States / cities: Aurora, Colorado • Atlanta, Georgia • New Orleans, Louisiana + 1 more

Conditions: Sickle Cell Disease, HEMOGLOBIN SS, Hemoglobin S Beta-0 Thalassemia, Inflammation, Quality of Life
Interventions: Omega-3 Fatty Acids: Eicosapentaenoic Acid (EPA) and Docosahexaenoic Acid (DHA), Placebo Capsules; Dietary Supplement · Other
Lead sponsor: Thomas Jefferson University; Other
Eligibility: 10 Years to 19 Years
Enrollment: 48 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2012
U.S. locations: 2
States / cities: Philadelphia, Pennsylvania

Conditions: Seizures
Interventions: Cannabidiol Oral Solution; Drug
Lead sponsor: INSYS Therapeutics Inc; Industry
Eligibility: 1 Year to 18 Years
Enrollment: 52 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2017
U.S. locations: 11
States / cities: San Francisco, California • Miami, Florida • Pensacola, Florida + 8 more

Conditions: Anemia, Sickle Cell
Interventions: Hydroxyurea; Drug
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 2 Years to 7 Years
Enrollment: 163 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2011
U.S. locations: 14
States / cities: Birmingham, Alabama • Washington D.C., District of Columbia • Miami, Florida + 10 more

Conditions: Genetic Disease, Genetic Syndrome
Interventions: Genetic Enrollees; Genetic
Lead sponsor: Nicklaus Children's Hospital f/k/a Miami Children's Hospital; Other
Eligibility: Up to 21 Years
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2070
U.S. locations: 1
States / cities: Miami, Florida

Conditions: Congenital Adrenal Hyperplasia
Interventions: Crinecerfont, Placebo; Drug
Lead sponsor: Neurocrine Biosciences; Industry
Eligibility: 2 Years to 17 Years
Enrollment: 103 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 22
States / cities: Birmingham, Alabama • Los Angeles, California • Orange, California + 19 more

Conditions: Down Syndrome, Congenital Heart Disease
Interventions: Stanford Binet Intelligence Scales, Fifth Edition, Vineland Adaptive Behavior Scales Third Edition, Peabody Picture Vocabulary Test, Fifth Edition, Expressive Vocabulary Test, Third Edition, Leiter International Performance Scale, Third Edition, Social Communication Questionnaire, Current Form, Aberrant Behavior Checklist, Repetitive Behavior Scale, Revised; Other
Lead sponsor: Carelon Research; Other
Eligibility: 60 Months to 155 Months
Enrollment: 273 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2022 – 2023
U.S. locations: 13
States / cities: Wilmington, Delaware • Washington D.C., District of Columbia • Atlanta, Georgia + 10 more

Conditions: Cystic Fibrosis
Interventions: Ivacaftor; Drug
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 2 Years and older
Enrollment: 33 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2015
U.S. locations: 12
States / cities: Birmingham, Alabama • Denver, Colorado • Atlanta, Georgia + 9 more

Conditions: Atopic Dermatitis
Interventions: EpiCeram Skin Barrier Emulsion, Desonide Cream 0.05%; Device · Drug
Lead sponsor: Promius Pharma, LLC; Industry
Eligibility: 3 Months to 12 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009
U.S. locations: 5
States / cities: Denver, Colorado • Chicago, Illinois • Winston-Salem, North Carolina + 2 more

Conditions: Tourette Syndrome
Interventions: Valbenazine, Placebo oral capsule; Drug
Lead sponsor: Neurocrine Biosciences; Industry
Eligibility: 6 Years to 17 Years
Enrollment: 81 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2019
U.S. locations: 48
States / cities: Sun City, Arizona • Little Rock, Arkansas • Rogers, Arkansas + 43 more

Conditions: Methylmalonic Acidemia
Interventions: hLB-001; Biological
Lead sponsor: LogicBio Therapeutics, Inc; Industry
Eligibility: 6 Months to 12 Years
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 4
States / cities: Atlanta, Georgia • Pittsburgh, Pennsylvania • Nashville, Tennessee + 1 more

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A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

Mucopolysaccharidosis Type II Natural History

R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas

Serum Brain Natriuretic Peptide Levels and Pulmonary Hypertension in Pediatric Sickle Cell Patients

Study of CAR T-Cells Targeting the GD2 With IL-15+iCaspase9 for Relapsed/Refractory Neuroblastoma or Relapsed/Refractory Osteosarcoma

NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease

Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A

Methadone in Pediatric and Adult Sickle Cell Patients

A Clinical Study to Evaluate the Safety and Efficacy of T92 in Pediatric Patients With Tourette Syndrome

Study of Safety and Efficacy of Antihemophilic Factor/Von Willebrand Factor Complex in Surgical Subjects With Von Willebrand Disease (vWD)

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort

A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)

Arginine Therapy for Sickle Cell Disease Pain

Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease

A Randomized Trial of LOVAZA in Pediatric Sickle Cell Disease (SCD)

Cannabidiol Oral Solution as an Adjunctive Treatment for Treatment-resistant Seizure Disorder

Long-Term Effects of Hydroxyurea in Children With Sickle Cell Anemia (The BABY HUG Follow-up Study)

Diagnostic Odyssey: Whole Genome Sequencing (WGS)

Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)

Congenital Heart Disease: Impact on Learning and Development in Down Syndrome (CHILD-DS)

Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation

Comparison of the Efficacy and Safety of Two Topical Creams for Pediatric Atopic Dermatitis

Safety and Efficacy of NBI-98854 in Pediatric Subjects With Tourette Syndrome

Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia